Last-Resort drug alpelisib made available for patients with no other options

NCT ID NCT04085653

AVAILABLE Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This program provides access to the drug alpelisib (BYL719) for patients with PIK3CA-related overgrowth spectrum or breast cancer who have no other treatment options and cannot join a clinical trial. It is not a study but a way for doctors to request the drug for individual patients with serious or life-threatening conditions.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: alpelisib (BYL719)
What this could lead to: If successful, this program could provide a treatment option for patients with serious diseases who have no other alternatives.
What could go wrong: This is not a clinical trial but a managed access program, so results are not systematically collected. The drug may not work for every patient and carries potential risks.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

breast cancer breast neoplasm hormone receptor-positive breast cancer PIK3CA-related overgrowth spectrum

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BREAST CANCER PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)

More trials for these conditions

Other studies related to the condition(s) this trial covers.